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Roche Reports Positive Evrysdi Data From Dose-Finding Part Of Spinal Muscular Atrophy Trial

  • Roche Holding AG (OTCMKTS: RHHBY) has reported positive data from the dose-finding Part 1 FIREFISH Phase 2/3 study of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA).
  • Evrysdi helps in the survival of motor neuron 2 (SMN2) splicing modifier designed for treating SMA by boosting SMN protein production. The protein is vital to maintaining healthy motor neurons and movement.
  • According to the latest data from 21 infant participants, Evrysdi treatment at 12 months helped 90% of infants survive without needing permanent ventilation, and 33% sit without support for at least five seconds.
  • Furthermore, it raised the survival levels of SMN protein by a median 1.9-fold from baseline in the high-dose group at 12 months.
  • In the low- and high-dose cohorts, no infant lost the ability to swallow over 12 months, and 86% were able to feed orally, either exclusively or in combination with a feeding tube at 12 months.
  • Price Action: RHHBF is up 1.03% $344 in market trading hours on the last check Thursday.

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